english.prescrire.org > Annual Prescrire Awards > The Prescrire Awards for 2021 > The Prescrire Drug Awards for 2021 > 2021 Drug Awards > Givlaari° (givosiran): on the Honours List at the 2021 Prescrire Drug Awards

The Prescrire Awards for 2021

The Prescrire Drug Awards for 2021

The Prescrire Drug Awards for 2021
Givlaari° (givosiran): on the Honours List at the 2021 Prescrire Drug Awards

Le Palmarès des Médicaments Drugs included on the Honours List constitute a clear advance for some patients compared with existing therapeutic options, albeit with limitations.

• GIVLAARI° (givosiran) – Alnylam

In acute hepatic porphyria in patients aged 12 years and over (Prescrire Int n° 227).

Givosiran in acute hepatic porphyria: fewer attacks, and no further attacks for some patients, at least in the short term.

Acute hepatic porphyrias are rare conditions, characterised by the accumulation of certain toxic precursors of haem, a molecule containing ferrous iron, present for example in haemoglobin. This provokes serious attacks, marked by severe abdominal pain, often accompanied by neurological and psychiatric disorders. These attacks constitute a medical emergency, and are sometimes fatal if untreated. The long-term complications are renal, hepatic and neurological.

Haem arginate is the standard treatment for attacks, and it is sometimes used off-label to prevent attacks. Preventive treatment with haem argenate is burdensome, requiring one to four intravenous infusions per month.

Givosiran (Givlaari°) is a "small interfering" ribonucleic acid (siRNA), which has been authorised in the prevention of acute hepatic porphyria attacks. In a double-blind, randomised, placebo-controlled trial in 94 patients, the estimated rate of attacks requiring hospitalisation, an urgent medical consultation or administration of haem arginate was 3 attacks per patient per year in the givosiran group, versus 12 attacks per patient per year in the placebo group. 50% of the patients in the givosiran group had no attacks during this 6-month trial, versus 17% in the placebo group.

Givosiran's main adverse effects are injection site reactions, hypersensitivity reactions, hepatic disorders and renal disorders, including renal failure. Givosiran is a more convenient preventive treatment than haem arginate, because it is administered once a month by subcutaneous injection.

These data earned givosiran a place on the 2021 Honours List. The possibility that givosiran prevents the long-term complications of the disease is a hypothesis that remains unproven as of late 2021.

A Pilule d'Or (Golden Pill) is granted to drugs that represent a major therapeutic advance in a field in which no treatment was previously available. Drugs included on the Honours List constitute a clear advance for some patients compared with existing therapeutic options, albeit with limitations. Drugs deemed "Noteworthy" provide a modest improvement in patient care.
SEE: > About the Prescrire Drug Awards for 2021

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 FREE  "The Prescrire Awards for 2021" Prescrire International 2022; 31 (235): 79-82. (pdf, free)


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